[Research advances on gene therapy for hemophilia].
作者信息
Dai X Y, Zhang L
机构信息
Institute of Hematology & Blood Disease Hospital, CAMS & PUMC, Tianjin 300020, China.
出版信息
Zhonghua Xue Ye Xue Za Zhi. 2018 Apr 14;39(4):350-352. doi: 10.3760/cma.j.issn.0253-2727.2018.04.022.
Abstract
摘要
相似文献
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[Research advances on gene therapy for hemophilia].[血友病基因治疗的研究进展]
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本文引用的文献
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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.采用高特异性活性因子IX变体的B型血友病基因疗法。
N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538.
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Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.具有修饰的弗林蛋白酶切割位点的新型凝血因子 VIII 变体可提高A型血友病基因治疗的疗效。
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CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse.CRISPR/Cas9介导的新型凝血因子IX基因突变的体细胞校正改善了小鼠血友病症状。
EMBO Mol Med. 2016 May 2;8(5):477-88. doi: 10.15252/emmm.201506039. Print 2016 May.
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Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas.人类肝细胞癌中反复出现的 AAV2 相关插入性突变。
Nat Genet. 2015 Oct;47(10):1187-93. doi: 10.1038/ng.3389. Epub 2015 Aug 24.
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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.FIX基因疗法治疗B型血友病的长期安全性和有效性
N Engl J Med. 2014 Nov 20;371(21):1994-2004. doi: 10.1056/NEJMoa1407309.
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The incidence of factor VIII inhibitors in severe haemophilia A following a major switch from full-length to B-domain-deleted factor VIII: a prospective cohort comparison.从全长因子VIII主要转换为B结构域缺失的因子VIII后，重度A型血友病患者中因子VIII抑制剂的发生率：一项前瞻性队列比较。
Haemophilia. 2015 Mar;21(2):219-226. doi: 10.1111/hae.12563. Epub 2014 Nov 7.
7
Oral gene therapy for hemophilia B using chitosan-formulated FIX mutants.壳聚糖制剂的 FIX 突变体用于乙型血友病的口服基因治疗。
J Thromb Haemost. 2014 Jun;12(6):932-42. doi: 10.1111/jth.12572.
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The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals.腺相关病毒衣壳的中和抗体在年轻的日本个体中减少。
J Med Virol. 2014 Nov;86(11):1990-7. doi: 10.1002/jmv.23818. Epub 2013 Oct 17.
9
Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice.通过慢病毒基因传递到造血干细胞进行血小板基因治疗可恢复 FIX(null) 小鼠的止血功能并诱导体液免疫耐受。
Mol Ther. 2014 Jan;22(1):169-77. doi: 10.1038/mt.2013.197. Epub 2013 Aug 23.
10
Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.接受 AAV 介导的基因转移 10 年后，严重血友病 B 患者骨骼肌中因子 IX 的表达。
Blood. 2012 Mar 29;119(13):3038-41. doi: 10.1182/blood-2011-09-382317. Epub 2012 Jan 23.
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